Gene therapy holds substantial promise for the treatment of a wide range of genetic and acquired diseases. Despite its potential, efficient and safe delivery of therapeutic genes into target cells remains a significant challenge. Lipoplexes, lipid-base formulations complexed with nucleic acids, have emerged as an attractive non-viral vector for gene delivery due to their ability to encapsulate and protect nucleic acids and facilitate their delivery into cells. This review focuses on the design, mechanism of action, and structure of lipoplexes for gene therapy and discusses the role of various lipids used in the formation of lipoplexes. Furthermore, we explore the application of lipoplexes in various disease models, cystic fibrosis, and cancers, highlighting recent advances. We conclude that while challenges remain, lipoplexes represent a promising strategy for safe and effective gene therapy.
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Hirlekar et al. (2026) studied this question.
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